Gene Editing Reverses Baby’s Leukemia

‘Gene Editing’ Reverses Baby’s Leukemia

Nov. 6, 2015 — Doctors in the United Kingdom have successfully treated a baby girl diagnosed with incurable leukemia by using a gene-editing technique that had been tested only in the laboratory.

The parents of 1-year-old Layla Richards from London gave permission for the experimental treatment when they learned doctors had exhausted all other options.

Experts at Great Ormond Street Hospital, where Layla was treated, say the little girl is “cancer free and doing well.”

Learn more about gene editing with these common questions.

What is the technique, exactly?

Gene editing is an exciting scientific advance that has the potential to treat a wide range of diseases caused by faulty genes.

It involves scientists basically making tiny molecular scissors that can cut and edit genes and reprogram cells.

The technique is officially still in the lab-testing phase. But if it can be perfected, it may have the potential to treat millions of people with genetic diseases.

What gene editing technique was Layla treated with?

Doctors used the “molecular scissors,” known as TALEN proteins, to edit genes and create designer immune cells programmed to hunt out and kill drug-resistant leukemia.

Because this treatment is at an early stage, it was sanctioned only because the girl had no other hope of surviving the disease — called acute lymphoblastic leukemia (ALL) — and her parents were willing to try it.

Doctors used modified T-cells from donors, known as UCART19 cells, to treat Layla.

“The approach was looking incredibly successful in laboratory studies,” says Professor Waseem Qasim, a consultant immunologist at the hospital. “And so when I heard there were no options left for treating this child’s disease, I thought, ‘Why don’t we use the new UCART19 cells?’”

“The treatment was highly experimental and we had to get special permissions, but she appeared ideally suited for this type of approach.”

Layla’s parents were also eager to try the treatment. “We didn’t want to accept palliative care, and so we asked the doctors to try anything for our daughter, even if it hadn’t been tried before,” says her mother, Lisa.

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